SMN2 and proximal spinal muscular atrophy: Providing multiple SMN2 ASO doses results in greater phenotypic amelioration in SMA mice (Hua et al., 2011; Zhou et al., 2015; Hammond et al., 2016), while increased concentrations of ASOs or AAVs have a similar positive effect (Meyer et al., 2015; Hammond et al., 2016; Hosseinibarkooie et al., 2016).